Medicines in Development for Older Americans - Backgrounder

Selected Medicines in Development for Older Americans

Alzheimer’s disease– A gene therapy in development for the treatment of Alzheimer's disease is designed to deliver nerve growth factor (NGF) to the brain. NGF is a naturally occurring protein that is important for neuron survival. The gene treatment is injected into a region in the brain where neuron degeneration occurs in Alzheimer’s disease. It is thought that the resulting sustained expression of NGF in the neurons can restore their lost function, leading to memory and cognition improvement. Another medicine in development is a humanized monoclonal antibody that is designed to bind to the beta-amyloid protein, drawing it away from the brain through the blood system. This process could reduce the accumulation of the protein, preventing the subsequent plaque formation that is seen in Alzheimer’s disease.

Chronic Obstructive Pulmonary Disease (COPD)– Several medicines in development for COPD are fixed-dose combinations of two or more medicines. One is a combination of two long-acting medicines with different biological actions. Both act to relax smooth muscles in the bronchial passages, leading to improved lung function for up to 24 hours, which allows for once-daily dosing.

Dementia – A dementia treatment in development selectively targets 5HT6 receptors in the brain. These receptors are associated with learning and memory. Currently available symptomatic treatments, such as cholinesterase inhibitors, are not brain-specific and, as a result, can alter systems in the entire body, leading to possible side effects. A targeted treatment like this has the potential to reduce systemic side effects.

Diabetes, Type 2– A potential first-in-class medicine in development for type 2 diabetes is a selective agonist of GPR40, a receptor expressed in pancreatic islet cells (a cluster of cells that produce hormones). Insulin, which breaks down glucose in the body to create energy, is secreted when glucose levels rise. This potential new medicine increases insulin secretion without causing insulin to significantly lower blood sugar.

Depression – A potential first-in-class treatment for major depression is in development based on the theory that chronic exposure to stress hormones may prevent the growth of new neurons in the brain, leading to depression and other conditions. The medicine, which can cross the blood-brain barrier, recruits the patients’ own neural stem cells to repair or protect against damage to the central nervous system.

Diabetic Kidney Disease – A potential first-in-class medicine is in development for the treatment of diabetic nephropathy – a chronic progressive kidney disease that is the leading cause of end-stage renal disease (ESRD) or kidney failure. This treatment may protect kidney function and slow disease progression when added to existing therapy.

Heart Failure – A genetically-targeted enzyme replacement therapy for heart failure is being tested to restore levels of a specific gene that promotes a failing heart to pump better and minimizes the severity of heart failure. In all forms of late-stage heart failure, levels of the gene decline, resulting in deficient heart function.

Osteoarthritis– Osteoarthritis is characterized by the breakdown of cartilage – the part of a joint that cushions the ends of the bones and allows easy movement. As cartilage deteriorates, bones begin to rub against one another, causing pain and difficult of movement. A potential first-in class medicine is in development that inhibits the gene encoding protein that plays a role in inflammation that causes pain in osteoarthritis.

Osteoporosis – An oral, once-weekly medicine is in development for osteoporosis. This treatment is an inhibitor of a protease enzyme that is involved in the metabolic breakdown of elastin, collagen and gelatin in the body, causing the deterioration of bone and cartilage.

Rheumatoid Arthritis (RA)– One medicine in development for RA is a fully human monoclonal antibody (mAb) directed against interleukin-6 (IL-6), a naturally occurring signaling protein involved in the regulation of immune and inflammatory responses associated with the disease. The mAb interrupts the inflammatory signaling cascade of IL-6 and has been shown in clinical trials to reduce the signs and symptoms of RA by targeting inflammation.